The drug discovery process is currently undergoing a fundamental revolution as it embraces “functional genomics”, that is, high throughput genome- or gene-based biology. This approach as a means to identify genes and gene products as therapeutic targets is rapidly superceding earlier approaches based on “positional cloning”. A phenotype, that is a biological function or genetic disease, would be identified and this would then be tracked back to the responsible gene, based on its genetic map position.
Functional genomics relies heavily on high-throughput DNA sequencing technologies and the various tools of bioinformatics to identify gene sequences of potential interest from the many molecular biology databases now available. There is a continuing need to identify and characterise further genes and their related polypeptides/proteins, as targets for drug discovery.
The amino acid γ-aminobutyric acid (GABA) is a dominant inhibitory neurotransmitter in brain and was found widely distributed throughout the nervous system. For the mouse homolog of the novel GABA transporter a developmentally regulated expression was determined. The mRNA of the mouse homolog NTG2 is abundant in neonatal brain. The NTG2 protein transports γ-aminobutyric acid, β-alanine and taurine (Liu QR et al (1993) J. Biol. Chem 268 (3), 2106–2112). The γ-aminobutyric acid (GABA) is a neurotransmitter involved in various central nervous system diseases.
Diseases to be treated including but are not limited to schizophrenia, epilepsy, depression, learning disorders, cognitive disorders, neurodegenerative diseases, multiple sclerosis, dementia, Alzheimers disease, Parkinsons disease, hyperactivity, anxiety disorder, sleeping disorder, alcoholism, muscular disorders (e.g. tremor), pain, headache, and migraine.